P763: EFFICACY AND SAFETY OF AVATROMBOPAG IN THE TREATMENT OF TRANSFUSION-DEPENDENT NON-SEVERE APLASTIC ANEMIA
Authors: Ting Zhang, Guangsheng He, Jinning Shi, Yuemin Gong, Yawen Zhang, Qiqiang Long, Jianyong LI
Journal: HemaSphere (2023)

Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: There no suitable definite treatment for transfusion-dependent non-severe aplastic anemia (TD-NSAA). Aims: This study aimed to evaluate the efficacy and safety of avatrombopag combined with CsA in patients with transfusion-dependent non-severe aplastic anaemia (TD- NSAA). Methods: From March 2021 to March 2023, 12 patients with TD- NSAA treated with CsA combined with avatrombopag in Nanjing Jiangning Hospital, Jiangsu Province Hospital and Second Hospital of Nanjing were enrolled. Avatrombopag was taken at a dose of 40 mg per day. CsA was initiated at 3~5 mg/kg daily and then adjusted according to side effects, to maintain the target concentration between 150 and 250 μg/L. Complete the blood counts，biochemical indexes and adverse events during avatrombopag treatment were recorded. The data was collected by prospective registration in the Chinese Eastern Collaboration Group of Anemia (ChiCTR2100045895). Results: Among the 12 patients with TD- NSAA, the median age was 58 (8-78) years, 5 were male and 7 were female (Table 1). Two patients did not respond to previous CsA combined with TPO-RA (eltrombopag/ haitrombopag). Three patients had abnormal hepatic function. The median duration of treatment with avatrombopag was 5.5 (2-20) months. Ten patients (83.3%) obtained trilineage HR [one cases of complete treatment response (CTR), four case of good treatment response (GPR), and four cases of partial treatment response (PR),one case of partial no treatment response (NR)]. Two patient was not included in the outcome assessment due to the short duration of treatment. The median interval time for patients to achieved response was 62 days （range 27-174 days）. White blood cell count（P=0.021）, and platelet count（P=0.003）,absolute neutrophil count(P=0.039),increased significantly after treatment with avatrombopag by pair test (Figure 1). Five patients achieved transfusion independent. Of the two TPO-RA (eltrombopag/ haitrombopag) converted patients, one obtained GPR, one patient PR. No avatrombopag-related grade 2 or higher adverse events, and no thrombotic events and hepatic or renal insufficiency occurred during avatrombopag treatment. Summary/Conclusion: This preliminary study showed that avatrombopag is an effective option for patients with transfusion-dependent non-severe aplastic anemia (TD- NSAA). Table 1 Comparison of data before and after the use of avatrombopag - Before avatrombopag therapy After avatrombopag therapy P value Gender, n (%) / Male 5 (41.67%) Female 7 (58.33%) Median age (range) (year) 58 (8, 78) / Classification, n (%) / complete treatment response (CTR) 1 (8.33%) Good treatment response (GPR) 4(33.33%) treatment response (PR) 4(33.33%) No treatment response (NR) 1(8.33%) WBC (×109/L) (range) 2.21 (1.12, 4.61) 3.88 (2.6,.17) 0.021 ANC (×109/L) (range) 0.92 (0.17, 0.88) 2.38 (1.06, 14.64) 0.039 RBC (×1012/L) (range) 2.05 (1.04, 3.12) 2.43 (1.33, 3.32) 0.312 Hb (g/L) (range) 81 (52, 128) 96(66, 126) 0.299 BPC (×109/L) (range) 11(1, 37) 48.5(21, 137) 0.003 RET (%) (range) 1.84(0.031,3.03) 2.44 (1.66, 3.79) 0.077 LDH (U/L) (range) 216(172, 271) 228 (179, 337) 0.153 Abbreviations: WBC, white blood cell count; ANC, absolute neutrophil count; RBC, red blood cell count; Hb, hemoglobin; BPC, blood platelet count; LDH, lactate dehydrogenase;RET reticulation red blood cells Figure 1 After avatrombopag therapy, the white blood cell count (2.21×109/L vs 3.88×109/L, p=0.021), absolute neutrophil count (0.92×109/L vs 2.38×109/L, p=0.039) and blood platelet count (11×109/L vs 48.5×109/L, p=0.003) improved significantly. Keywords: Treatment, Aplastic anemia, Cyclosporin A, TPO